Abstract
BACKGROUND/OBJECTIVES: In early-stage oncology clinical trials, the use of endpoints beyond overall survival (OS), including recurrence-free survival (RFS) or event-free survival (EFS), is becoming more common. To understand whether these outcomes are important to patients, this study explored the perceived value of non-OS endpoints among Canadians treated for early-stage cancer or with curative intent. METHODS: Canadians treated for early-stage breast, lung, or gastrointestinal cancer participated in semi-structured interviews. Participants provided perspectives on OS, RFS, disease-free survival (DFS), EFS, and pathological complete response (pCR) endpoints. Reflexive thematic analysis was used to explore patterns in responses and alignment of trial endpoints with patient treatment goals, priorities and preferences. RESULTS: The mean age of the 33 participants was 54.8 years, and 21 were female; 28 reported prior surgery, and 21 were also treated with chemotherapy (11 specified as neo-adjuvant; 9 specified adjuvant). All participants valued OS, and most viewed non-OS endpoints as reflective of their treatment priorities, including maintaining health-related quality of life and getting back to 'normal'. They also valued timely and equitable treatment access and equated having access to new treatments with better options. While participants considered efficacy data from clinical trials provided by non-OS endpoints sufficient to want access to new treatments, the relative importance of being disease- or recurrence-free versus maximizing length of life differed according to recurrence status, prognosis, cancer type and life stage. CONCLUSIONS: These findings support the relevance and importance of non-OS endpoints to Canadians with early-stage cancer and highlight participants' desire for rapid approval of treatments with demonstrated improvements in non-OS endpoints.